Rare Disease Report

New Study Testing Genetically Modified Skin Cells Being Injected Into EB Wounds

JUNE 08, 2017
James Radke
The first part of a Phase 1/2 clinical trial that evaluates the feasibility of injecting genetically modified skin cells to produce collagen in wounds of recessive dystrophic epidermolysis bullosa (RDEB) patients has completed enrollment.
 
RDEB, the most severe form of epidermolysis bullosa (EB), is caused by a mutation of the COL7A1 gene, which encodes for collagen VII. The study by Fibrocell is introducing the gene into these patients.
 
EB patients have extremely delicate skin, often referred to as “butterfly skin,” because the skin becomes fragile – like the wings of a butterfly – and can fall apart at the touch. The disorder makes patients susceptible to blisters and poor healing of wounds and more than 50,000 people worldwide suffer from it.
 
The primary objective of this open-label clinical trial is to evaluate the safety of FCX-007 but additional outcome measures will also be assessed.
 
All 6 patients received their initial dosing of FCX-007, autologous fibroblasts genetically modified to produce functional collagen VII. The fibroblasts are taken from the patient and infused with the gene to produce collagen VII before being cultured and frozen. When ready for use, they are thawed and injected into the skin near the wound.
 
As a control, similar wounds on the patients are monitored over the course of the 52-week study, but the company hopes to provide a 12-week update in the third quarter of 2017. 
 
All patients in the current trial are adults. In the second phase of the trial, 6 children with RDEB will be given FCX-007. Prior to conducting that pediatric trial, the U.S. Food and Drug Administration (FDA) is requiring that evidence be submitted showing the therapy is safe and beneficial. 
 
To learn more about the clinical trial, visit www.clinicaltrials.gov/ct2/show/NCT02810951
 
There are currently several treatment options in development for EB, and Abeona is another company using genetically modified fibroblasts to treat RDEB. Unlike FCX-007, in which fibroblasts are injected into the wound, the genetically modified fibroblasts by Abeona, called EB-101, are on skin grafts that are placed on the wound.


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